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Novel hybrid silicon-lipid nanoparticles deliver a siRNA to cure autosomal dominant osteopetrosis in mice. Implications for gene therapy in humans.
Maurizi A, Patrizii P, Teti A, Sutera FM, Baran-Rachwalska P, Burns C, Nandi U, Welsh M, Torabi-Pour N, Dehsorkhi A, Saffie-Siebert S. Maurizi A, et al. Among authors: saffie siebert s. Mol Ther Nucleic Acids. 2023 Aug 19;33:925-937. doi: 10.1016/j.omtn.2023.08.020. eCollection 2023 Sep 12. Mol Ther Nucleic Acids. 2023. PMID: 37680985 Free PMC article.